E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Growth Hormone Deficiency |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10056438 |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of the study is to assess the safety and tolerability of ACP-001 under multiple-dose conditions in adult patients with Growth Hormone Deficiency |
|
E.2.2 | Secondary objectives of the trial |
• To assess the pharmacokinetics of three different dose-levels of ACP-001 over a 4-week course of treatment • To assess the pharmacodynamic response of three different dose-levels of ACP-001 and the active comparator, daily Human Growth Hormone, over a 4-week course of treatment |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female between 20 to 70 years 2. Body Mass Index (BMI, kg/m2) of 19.0 to 36.0 kg/m2, both inclusive 3. Adult Growth Hormone Deficient (AHGD) patients with documented growth hormone deficiency as defined in the Consensus guidelines for the diagnosis and treatment of adults with GH deficiency II (Consensus Guidelines 1998 and 2007) 4. Fertile females must agree to use appropriate contraceptive methods and have a negative pregnancy test at inclusion 5. GH replacement therapy for at least 3 months 6. Willing to maintain current activity level during the trial 7. Subjects are able and willing to provide written informed consent and authorization for protected health information disclosure in accordance with Good Clinical Practice (GCP) |
|
E.4 | Principal exclusion criteria |
Any subject who meets any of the following criteria will not qualify for entry in the study: 1. History of hypersensitivity and/or idiosyncrasy to any of the test compounds or excipients employed in this study. 2. Females of childbearing potential who are pregnant, breast-feeding or intend to become pregnant or are not using adequate contraceptive methods. Reliable methods for women are orally administered hormonal contraceptives, surgical intervention (e.g. tubal ligation), intrauterine device (IUD) and sexual abstinence. 3. Active malignant disease or malignant disease within the last 5 years 4. Proliferative retinopathy judged by retina-photo within the last year 5. Heart insufficiency as judged by the investigator and/or NYHA 3 or greater (NYHA criteria for diagnosis of diseases of the heart, 1994) 6. Subjects with uncontrolled diabetes with an HbA1c above 8.0% and/or insulin treatment 7. Stable pituitary hormone replacement therapy for less than 3 months 8. Impaired liver function as judged by the investigator or hepatic transaminases > 2 times the upper limit of normal 9. Impaired kidney function as judged by the investigator and/or creatinine clearance <50 mL/min and/or serum creatinine > 1.4 mg/dL 10. Participation in another interventional clinical study involving an investigational compound within 3 months prior to enrolment in this study or participation in another interventional clinical study involving an investigational compound during this study. 11. Subjects who are unable to comply with the requirements of the study or who in the opinion of the investigator should not participate in the study. 12. History or presence of alcohol abuse or drug abuse. 13. Patients with known history for, or presence of, anti-hGH and / or anti-PEG antibodies |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Pharmacokinetics (PK): •Change in TransCon PEG hGH serum concentration from baseline to last treatment period •Change in hGH serum concentration from baseline to last treatment period Efficacy/Pharmacodynamics (PD): •Primary efficacy: The mean absolute and percent change of plasma IGF-I from baseline to last treatment period •Secondary efficacy: The mean absolute and percent changes in plasma IGFBP-3 from baseline to last treatment period; alterations in GHBP from baseline to last treatment period Safety and Tolerability: Adverse events, injection site reactions, physical examinations, laboratory measurements, with particular emphasis on changes in alanine aminotransferase (ALT), aspartate aminotransferase (AST), blood glucose, immunogenicity (anti-hGH antibodies; anti-PEG antibodies) and injection site reactions. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Laboratorio parzialmente in cieco |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |